Riassunto analitico
Cholesterol plays a crucial role in major cardiovascular and neurodegenerative diseases, including some rare genetic disorders showing altered cholesterol metabolism such as Niemann-Pick Type C. Cyclodextrins (CDs) have shown promising therapeutic efficacy based on their capacity to sequester and mobilise cholesterol. The functionalisation of CDs onto SPIONs could represent an attractive theranostic platform to enhance the delivery of the cholesterol-mopping macrocycle in order to provide improved therapy for neurodegenerative disorders. Through a process of receptor-mediated accumulation, CD-based, pH-responsive superparamagnetic iron oxide nanoparticles (SPIONs) will release CDs specifically into the lysosome where the cholesterol-mopping activity is most required.
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